Expanded Access Navigator FAQs

1. I have an emergency situation; access needed within hours or days. Who do I call once I have manufacturer agreement?

If your patient is in a situation where access is needed in a matter of hours or days, and a company is willing and able to provide the product needed, FDA can be contacted directly through its Division of Drug Information at 855-543-3784 or druginfo@fda.hhs.gov for electronic or over-the-phone application processing. To request emergency access to investigational biological products regulated by CBER, call 240-402-8010 or 800-835-4709 or industry.biologics@fda.gov.

2. Can patients or physicians apply for expanded access to investigational therapies directly from the Foundation site?

No, the site is designed as a clearinghouse for information and resources. Requests for expanded access go through a multi-site review process involving pharmaceutical companies, the FDA and Institutional Review Boards to ensure that potential benefits of unapproved compounds are weighed against the risks. The Expanded Access Navigator collects expanded access policies in one place and guides physicians, patients and their caregivers through the steps involved in this complex process.

3. What factors do companies consider?

You can find each companies full expanded access criteria on the directory of links, but most take into account the severity of the patient’s condition, whether alternate approved therapies exist, whether risks are acceptable, if the supply of the product is adequate and whether expanded access permission will delay the development of the product for its intended users.

4. If a company lists its expanded access policy on this site, am I guaranteed it will accept my request?

There are conditions under which a company will not be able to grant access to its products, and even if it does your doctor will need to work closely with the FDA and an Institutional Review Board for final approval. But the steps are clearly outlined in the Navigator to help clarify the process.

5. If approved, is expanded access free to patients?

Many pharmaceutical companies work with patients who can’t afford the treatments, but policies vary and insurance plans might not cover costs of investigative therapies granted under expanded access.

6. Are physicians compensated for the time involved in making a request?

Not always and since it is a time-consuming process many physicians must also seek administrative support when pursuing expanded access.

7. I’m not eligible for a clinical trial of an investigational therapy, are there other options for expanded access?

There are two other options. You might be eligible for an existing intermediate or large size expanded access programs called EAPs, which are listed under the ClinicalTrials.gov link. You might also be eligible for single-patient expanded access, which is always requested by a treating physician on behalf of the patient.

8. Are companies required to offer Single-Patient Expanded Access?

No, but new legislation does require companies to make their policies concerning single-patient expanded access available to the public. That information can be researched through the links to company EA policies in the Navigator directory.

9. Does FDA have the final say?

The FDA is the first step in the request process in emergency cases but non-emergency requests for single-patient expanded access first go from the patient’s physician to the pharmaceutical company. If the company agrees to provide a drug and provides a Letter of Authorization then the FDA reviews the case. Institutional Review Boards also have a voice in the approval process, but once those independent requirements have been met the FDA approves the vast majority (99%) of single-patient expanded access requests within 15 days.

10. Identifying a rare disease often takes years; can I apply for expanded access without a diagnosis?

For single patient expanded access beyond trials, a criterion commonly required by companies is that the patient has exhausted standard treatments. While this may be difficult to demonstrate without a clear diagnosis, it is not impossible. Start with ClinicalTrials.gov and read the expanded access policies voluntarily supplied by companies on the Navigator’s Directory.

11. What counts as a serious or life-threatening disease, especially if it's rare or undiagnosed?

This is an important topic to discuss with your referring or treating physician because to apply for expanded access your disease or condition must be either serious or life-threatening. “Serious” is a clinical judgement based on many factors but can be summarized as having a substantial impact on day-to-day functioning. “Life-threatening” means there is a reasonable likelihood of death within months or prematurely without early treatment.

12. A majority rare diseases, 80%, are genetic; can I apply for expanded access for investigational gene therapies?

Yes, gene therapies and other biologics are types of investigational treatments available for patients with serious or life-threatening conditions, who are unable to participate in clinical trials and have exhausted other treatment options.

13. I want a drug that's been taken off the market for safety reasons; can I get it through expanded access?

According to FDA guidance updated in 2017, expanded access may also refer to (1) use in situations when a drug has been withdrawn for safety reasons, but there is a patient population for whom the benefits of the withdrawn drug continue to outweigh the risks; (2) use of a similar, but unapproved drug (e.g., foreign-approved drug product) to provide treatment during a shortage of the approved drug; (3) use of an approved drug where availability is limited by a risk evaluation and mitigation strategy (REMS) by patients who cannot obtain the drug under the REMS; or (4) use for other reasons. Expanded access is sometimes requested in other situations, for example when an approved drug exists for the adult population but is requested for pediatric use. Drug company approval of expanded access requests for approved drugs is less common than for investigational therapies and costs and reimbursements vary.

14. I want a drug that is already on the market but not approved for my disease; can I get it through EA?

The Navigator is designed to focus on expanded access – access to drugs not yet approved by FDA. This website does not provide guidance on access to “off-label” treatments, because only your physician can provide medical guidance about uses that are not approved by FDA. Your physician can prescribe medications that the physician and patient agree are appropriate, even if that use is “off-label." Patients and physicians must make these decisions based on each medical situation, taking account of all relevant factors for individual patients.